Stem cell or bone marrow transplants

Bone marrow is a spongy material inside the bones which produces blood cells from stem cells. Stem cells are immature cells that develop into mature blood cells which are then released into the bloodstream. A stem cell or bone marrow transplant allows higher doses of chemotherapy to be used than usual, which may improve the chances of curing some types of leukaemia or lengthening remission. Very high doses of chemotherapy, sometimes with radiotherapy (Total Body Irradiation), are given to destroy the leukaemic cells in the bone marrow. The marrow and immune system are then ‘rescued’ with an infusion (drip) of stem cells either from the patient (autologous treatment) or from a donor (allogeneic transplant). Some types of allogeneic transplant use less chemotherapy and radiotherapy than an autologous transplant, and are known as reduced intensity transplants.

Stem cell transplants are complicated and risky procedures producing many side effects and possible complications. Patients remain in hospital isolation for at least a month afterwards, during which time they are vulnerable to life-threatening infections and at times will feel very ill. Transplants are offered only to people who are considered strong enough to withstand the physical and emotional demands. Reduced intensity transplants has expanded transplant options for older patients and those with co-morbidities. The age of patients considered for transplants is slowly increasing and a small number of patients over the age of 70 are now receiving allogeneic transplants.

Finding a donor
Having reduced the leukaemia to the lowest possible level using standard chemotherapy (see ‘Chemotherapy and how it is given’) and/or radiotherapy (see ‘Radiotherapy’), stem cells are collected from the patient or donor. Where possible, brothers and sisters (siblings) are tested for their suitability to be a donor as they are likely to be the best match. There is one chance in four of finding a suitable match among siblings. Parents are not usually a good match but may be tested if no other donor is found. When no relatives are suitable an unrelated match will be sought via the National Blood Transfusion Service or charity registers such as the Anthony Nolan Trust or cord blood stem cell transplants can be used where stem cells collected from the umbilical cords of newborn babies are transplanted into the patient..

Bone marrow transplant used to be the best chance of cure for patients with CML. Since around 2000, treatment with imatinib (Glivec) or similar drugs has resulted in a dramatic change in the outlook for people with CML so that transplant is now only used for a minority of patients (see ‘Biological therapies’). Several people we interviewed who were diagnosed with CML before imatinib (Glivec) became widely available were considered for a transplant. When no donor could be found for Chanelle her doctors were initially unsure how to treat her chronic myeloid leukaemia (CML). She was then invited to take part in a trial of a new drug, imatinib (Glivec), which she has taken ever since. No donor could be found for another woman with CML but after obtaining a second opinion it was agreed that a transplant was unnecessary. Despite her brother and sister both being a match, a third woman with CML decided not to have a stem cell transplant and obtained imatinib (Glivec) before it was routinely available in the UK. Frances, who had ALL, had cut her ties with her family so didn't want them to be contacted about bone marrow donation, but in the event she didn't need a transplant. Eight years after her chronic lymphocytic leukaemia (CLL) diagnosis, it was suggested that Beverley might need a stem cell transplant in the future so her siblings were tested but none were a match.

Some people were lucky to have a perfectly matched sibling. Both Deb and Aley had a brother in the Indian subcontinent who travelled to the UK to donate their stem cells. One of Elizabeth’s brothers was initially thought to be a match but this was later discovered to be a mistake. Her mother insisted on being tested and, unusually, was a perfect match. Mark’s mother was a partial match and her stem cells were frozen because his immediate need for a transplant receded. Some people with no suitable relatives found a match with an unrelated donor; Ann had a choice of two.

Harvesting stem cells
Stem cells are harvested (taken) from patients and donors in two ways. Some had liquid bone marrow drawn into a syringe by inserting a needle into the hip bone (pelvis). This is done under a general anaesthetic so people we spoke to found it uneventful, but felt bruised and sore afterwards. The cells are mixed with preservative and frozen until needed.

More commonly people are given injections of a growth factor (G-CSF) to stimulate production of stem cells and make them spill over into the bloodstream, followed by extraction via a cannula using a cell separator machine. Most people spend about four hours connected to the machine through a cannula, but for some it took longer or the procedure was repeated on a second day to obtain enough cells. Some people said the G-CSF injections stung. Mark said that a nurse injected the G-CSF too quickly the first time causing painful bleeding. One man’s wife did the injections for him at home. He was disappointed when his harvest was cancelled because there were not enough stem cells in his blood.

Stem cell harvesting is followed by the so-called ‘conditioning regimen’ of high dose chemotherapy with or without total body irradiation (TBI) given to the patient over a few days. High dose chemotherapy is given intravenously via a central line, like standard chemotherapy (see ‘Chemotherapy and how it is given’. For details of TBI see 'Radiotherapy').

Conditioning regimens cause unpleasant side-effects such as hair loss, nausea and vomiting, diarrhoea and mouth ulcers (see ‘Unwanted effects of treatments’) and usually leave the patient infertile. Two people had a reduced intensity transplant, which uses less chemotherapy or radiotherapy than usual with the aim of reducing transplant-related complications. Reduced intensity transplants are not suitable for everyone.

Transplanting stem cells
After the conditioning regimen the stem cells are given to the patient through a drip in the same way as a blood transfusion. It usually takes only a short time. People found this very quick and an anti-climax after the preparation. Elizabeth had an allergic reaction to the preservative.

During the first few weeks after the transplant, the people we spoke to had been very poorly and remembered little of the experience. Julie remembered 'doctors coming in and out and people cleaning me but other than that it’s just a total blur’.

*AML – Acute myeloid leukaemia

Last reviewed: December 2018.
Last updated: December 2018.

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